Study to Assess Long-Term Outcomes of Trientine in Wilson Disease Patients Withdrawn From Therapy With d-Penicillamine

NCT02426905 | Unknown Phase 4

• 1 other identifier: UNV-TRI-002
• Study Type: interventional
• Target: 90
• Locations: 4 countries
• Sites: 4

Brief Summary

A study to review Wilson disease patients who have previously been prescribed d- Penicillamine but were changed to trientine as treatment for their disease, and to follow them for a further 12 months.

Conditions

Wilson Disease

Outcome Measures

Primary Outcomes (2)

• Clinical outcome specific to the retrospective part of the study

  The clinical course of neurological and hepatic disease for each available time point after initiation of treatment (6, 12, 24, 36, and 48 months, and at the last available time point while taking second line trientine) will be scored (Investigator's score) based on neurological and hepatic status at the time of initiating trientine as: 1 = Unchanged 2 = Improved but not normal 3 = Improved to normal 4 = Asymptomatic over duration of therapy 5 = Worsened.

  48 months

• Clinical outcome specific to the prospective part of the study

  The clinical course of neurological and hepatic disease will be scored (Investigator's score) based on the status at 6 and 12 months after Baseline as: 1 = Unchanged 2 = Improved but not normal 3 = Improved to normal 4 = Asymptomatic over duration of therapy 5 = Worsened A patient will be counted as a responder if they have a rating of ≤4 at the 12 month visit for both the neurological and hepatic Investigator's score. They will be counted as a non-responder if they have a rating = 5 for one or both scores at the 12 month visit or if they were discontinued from the study for any reason prior to the 12 month visit.

  12 months

Secondary Outcomes (2)

• Safety Endpoint Applicable to both the Retrospective and Prospective Parts of the Study

  Up to 60 months

• Quality of Life Endpoints for the Prospective Part of the Study

  12 months

Study Arms (2)

Retrospective

NO INTERVENTION

Prospective

OTHER

Drug: trientine dihydrochloride

Interventions

trientine dihydrochloride DRUG

A retrospective review of patients' medical records

Prospective

Eligibility Criteria

• Age: 1 Year - 90 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Patients aged 1 year to 90 years of age.
• Physician established diagnosis of Wilson disease based on a Ferenci score \> 3.
• Documented treatment with d-Penicillamine, withdrawal of treatment with d- Penicillamine, followed by treatment with trientine for at least 6 months at date of informed consent.
• Able/willing to provide written informed consent.
• For enrolment in the prospective part, enrolment in the retrospective part of the study is required.

You may not qualify if:

• Incomplete history of medication use for trientine from initial diagnosis to latest follow up.
• Unavailable outcome data for hepatic and neurological course of disease at assessment time points.
• Patients with acute liver failure and fulminant hepatic disease with fatal outcome.
• Hypersensitivity to trientine and severe anaemia.

Sponsors & Collaborators

• Univar BV: lead

Study Sites (4)

Universitätsklinik Heidelberg

Heidelberg, 69120, Germany

Location

"Aghia Sophia" Children's Hospital

Goudi, 11527, Greece

Location

San Paolo Hospital UOC

Milan, 20142, Italy

Location

Kings College Hospital

London, United Kingdom

Location

Related Publications (2)

• Mohr I, Kruse C, Aliane V, Weiss KH. Prospective Study to Assess Long-Term Outcomes of Chelator-Based Treatment With Trientine Dihydrochloride in Patients With Wilson Disease. JGH Open. 2025 Mar 17;9(3):e70114. doi: 10.1002/jgh3.70114. eCollection 2025 Mar.

  PMID: 40104015 DERIVED

• Weiss KH, Kruse C, Manolaki N, Zuin M, Ferenci P, van Scheppingen D, Wijnberg L, de Koning CE, Dhawan A. Multicentre, retrospective study to assess long-term outcomes of chelator based treatment with trientine in Wilson disease patients withdrawn from therapy with d -penicillamine. Eur J Gastroenterol Hepatol. 2022 Sep 1;34(9):940-947. doi: 10.1097/MEG.0000000000002387. Epub 2022 Apr 29.

  PMID: 35482910 DERIVED

MeSH Terms

Conditions

Hepatolenticular Degeneration

Interventions

Trientine

Condition Hierarchy (Ancestors)

Liver Diseases; Digestive System Diseases; Basal Ganglia Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Movement Disorders; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Metabolism, Inborn Errors; Metal Metabolism, Inborn Errors; Metabolic Diseases; Nutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

Ethylenediamines; Diamines; Polyamines; Amines; Organic Chemicals

Study Officials

• Karl-Heinz Weiss, MD

  Universitätsklinik Heidelberg

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 18, 2014
• First Posted: April 27, 2015
• Study Start: January 1, 2016
• Primary Completion: May 1, 2018
• Study Completion: July 1, 2018
• Last Updated: August 23, 2017

Record last verified: 2017-08

Locations

IT (1); GR (1); GB (1); DE (1)